Immune Thrombocytopenia (ITP)Phase 1/2NCT07196163
An Exploratory Study of Golidocitinib in Adult Patients With ITP
A Phase 1/2 study testing Golidocitinib in people with Immune Thrombocytopenia (ITP), run by Peking University People's Hospital at 1 site in China.
Trial summary
This is a multicenter clinical study to evaluate the safety and efficacy of golidocitinib in patients with primary immune thrombocytopenia (ITP). The study consists of two parts: Part A dose escalation and Part B dose expansion. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Immune Thrombocytopenia (ITP)
- What is being tested?
- Golidocitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 138 participants
- Where
- 1 siteChina
- Study dates
- 30 Oct 2025to 30 Oct 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female participants between 18 and 80 years old.
- Primary ITP for >3 months.
- An average of two platelet counts (⩾ 7 days apart) of < 30 × 10⁹/L.
- Have relapsed or treatment-resistant to previous ITP therapies, including corticosteroids and at least one other ITP therapy.
- Have history of response to previous treatments.
- Adequate hematologic, hepatic, and renal fuction.
- Participants willing to comply with contraceptive restrictions.
Who cannot join
- Diagnosed with secondary immune thrombocytopenia, or there is evidence that the patient has a secondary cause of immune thrombocytopenia.
- Previous or current therapy and comedications meet exclusion criteria.
- Patients with major caridiovascular disease, active infetion, maligancy or uncontrolled systemic disease.
- Women who are breast feeding.
- History of hypersensitivity to sudy drug with a similar chemical structure or class.
- Previously received JAK inhibitors.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, Beijing Municipality, 100010
About this study
Official title: An Exploratory Clinical Study of Golidocitinib in Adult Patients With Primary Immune Thrombocytopenia (ITP)
This is a multicenter clinical study to evaluate the safety and efficacy of golidocitinib in patients with primary immune thrombocytopenia (ITP). The study consists of two parts: Part A dose escalation and Part B dose expansion. Part A is designed to obtain the safety profile of golidocitinib in patients with ITP and the recommended dose for the randomized cohort in Part B. Part B is a randomized, double-blind, placebo-controlled study, and the primary objective of this part is to evaluate the preliminary efficacy of golidocitinib in patients with ITP.
- Conditions
- Immune Thrombocytopenia (ITP)
- Being tested
- Golidocitinib, Placebo
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GolidocitinibDrug
Golidocitinib will be administered orally as capsules in a 28-day cycle.
PlaceboDrug
Placebo will be administered orally as capsules in a 28-day cycle.
| Study group | Role | What they receive |
|---|---|---|
| Golidocitinib Part B | Experimental | Golidocitinib at recommended phase 2 doses, administered orally, once daily. Receives: Drug: Golidocitinib |
| Placebo Part B | Placebo comparator | Placebo at recommended phase 2 doses, administered orally, once daily. Receives: Drug: Placebo |
| Golidocitinib Part A | Experimental | Golidocitinib dose escalation, administered orally, once daily. Receives: Drug: Golidocitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Incidence and severity of Adverse Events (AEs)
The severity was graded based on the National Cancer Institute's Common Terminology Criteria for Adverse Events.
Measured: 1 year
Part B: Durable response rate
The proportion of patients with a platelet count ≥ 50 × 10⁹/L on at least four of six scheduled visits between weeks 14 and 24
Measured: up to 24 weeks
Secondary outcomes (12)
Part A: Durable response rate
The proportion of patients with a platelet count ≥ 50 × 10⁹/L on at least four of six scheduled visits between weeks 14 and 24
Measured: up to 24 weeks
Part B: Incidence and severity of Adverse Events (AEs)
The severity was graded based on the National Cancer Institute's Common Terminology Criteria for Adverse Events.
Measured: 1 year
Part A and Part B: Overall response rate
The proportion of patients with a platelet count ≥ 100 × 10⁹/L on 2 consecutive visits at least 7 days apart and without bleeding
Measured: up to 24 weeks
Part A and Part B: Complete response rate
The proportion of patients with a platelet count ≥ 100 × 10⁹/L on 2 consecutive visits at least 7 days apart and without bleeding
Measured: up to 24 weeks
Part A and Part B: Early response rate
The proportion of patients with a platelet count ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding within 1 week
Measured: up to 1 week
Part A and Part B: Initial response rate
The proportion of patients with a platelet count ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding within 4 weeks
Measured: up to 4 weeks
Part A and Part B: Sustained response rate
The proportion of patients with a platelet count ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding between weeks 20 and 24
Measured: up to 24 weeks
Part A and Part B: Time to response
Time to first platelet counts ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding
Measured: up to 24 weeks
Part A and Part B: Cumulative response time
Number of cumulative days with platelet counts ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding by Week 24
Measured: up to 24 weeks
Part A and Part B: The proportion of patients requiring rescue therapy
Measured: up to 24 weeks
Part A and Part B: The proportion of patients experencing bleeding (≥ Grade 2)
Measured: up to 24 weeks
Part A and Part B: Changes of ITP-BAT bleeding score
Measured: up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2025
- Study start30 Oct 2025 (estimated)
- Primary completion30 Oct 2028 (estimated)
- Study completion30 Oct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- Part A-Open-label Sequential, Part B-Double-blind Parallel
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 138 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao Hui Zhang, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07196163
- Sponsor's protocol ID
- DZ2024J0003
- Status last verified
- Sep 2025
- First submitted
- 19 Sep 2025
- Last updated
- 29 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Clinical Study of Golidocitinib in Adult Patients With Primary Immune Thrombocytopenia (ITP)
- ClinicalTrials.gov ID
- NCT07196163
- Acronym / sponsor ID
- DZ2024J0003
- Conditions
- Immune Thrombocytopenia (ITP)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 138 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 30 Oct 2025 (estimated)
- Primary completion
- 30 Oct 2028 (estimated)
- Study completion
- 30 Oct 2029 (estimated)
- First posted
- 29 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter clinical study to evaluate the safety and efficacy of golidocitinib in patients with primary immune thrombocytopenia (ITP). The study consists of two parts: Part A dose escalation and Part B dose expansion. Part A is designed to obtain the safety profile of golidocitinib in patients with ITP and the recommended dose for the randomized cohort in Part B. Part B is a randomized, double-blind, placebo-controlled study, and the primary objective of this part is to evaluate the preliminary efficacy of golidocitinib in patients with ITP.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- Part A-Open-label Sequential, Part B-Double-blind Parallel
| Arm | Type | Receives |
|---|---|---|
| Golidocitinib Part B | Experimental | Drug: GolidocitinibGolidocitinib at recommended phase 2 doses, administered orally, once daily. |
| Placebo Part B | Placebo comparator | Drug: PlaceboPlacebo at recommended phase 2 doses, administered orally, once daily. |
| Golidocitinib Part A | Experimental | Drug: GolidocitinibGolidocitinib dose escalation, administered orally, once daily. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male and female participants between 18 and 80 years old.
- Primary ITP for >3 months.
- An average of two platelet counts (⩾ 7 days apart) of < 30 × 10⁹/L.
- Have relapsed or treatment-resistant to previous ITP therapies, including corticosteroids and at least one other ITP therapy.
- Have history of response to previous treatments.
Key exclusion criteria (6 listed)
- Diagnosed with secondary immune thrombocytopenia, or there is evidence that the patient has a secondary cause of immune thrombocytopenia.
- Previous or current therapy and comedications meet exclusion criteria.
- Patients with major caridiovascular disease, active infetion, maligancy or uncontrolled systemic disease.
- Women who are breast feeding.
- History of hypersensitivity to sudy drug with a similar chemical structure or class.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GolidocitinibGolidocitinib will be administered orally as capsules in a 28-day cycle. | Drug | Golidocitinib Part A, Golidocitinib Part B |
| PlaceboPlacebo will be administered orally as capsules in a 28-day cycle. | Drug | Placebo Part B |
Primary outcome
Part A: Incidence and severity of Adverse Events (AEs)
Time frame: 1 year
The severity was graded based on the National Cancer Institute's Common Terminology Criteria for Adverse Events.
Part B: Durable response rate
Time frame: up to 24 weeks
The proportion of patients with a platelet count ≥ 50 × 10⁹/L on at least four of six scheduled visits between weeks 14 and 24
12 secondary outcomes
Part A: Durable response rate
Time frame: up to 24 weeks
Part B: Incidence and severity of Adverse Events (AEs)
Time frame: 1 year
Part A and Part B: Overall response rate
Time frame: up to 24 weeks
Part A and Part B: Complete response rate
Time frame: up to 24 weeks
Part A and Part B: Early response rate
Time frame: up to 1 week
Part A and Part B: Initial response rate
Time frame: up to 4 weeks
Part A and Part B: Sustained response rate
Time frame: up to 24 weeks
Part A and Part B: Time to response
Time frame: up to 24 weeks
Part A and Part B: Cumulative response time
Time frame: up to 24 weeks
Part A and Part B: The proportion of patients requiring rescue therapy
Time frame: up to 24 weeks
Part A and Part B: The proportion of patients experencing bleeding (≥ Grade 2)
Time frame: up to 24 weeks
Part A and Part B: Changes of ITP-BAT bleeding score
Time frame: up to 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Golidocitinib in people with Immune Thrombocytopenia (ITP), run by Peking University People's Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 29 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part A: Incidence and severity of Adverse Events (AEs) (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2025.